Mice injected with spinal fluid from ALS patients offer potential therapeutic target

By The Science Advisory Board staff writers

August 23, 2022 -- Researchers at the Tisch MS Research Center of New York have shown in a novel animal model that by injecting mice with cerebrospinal fluid (CSF) from patients with the most common form of amyotrophic lateral sclerosis (ALS) they induced permanent motor disability associated with the pathological features of this aggressive and fatal chronic disease.

Their findings, published August 22 in the journal Brain Communications, suggest that an unlikely protein -- apolipoprotein B-100 -- is the culprit behind the kind of neurological degeneration that is a hallmark of ALS.

The paper seems to provide initial proof that filtering apolipoprotein B-100 out of CSF could improve symptoms of sporadic ALS, which occurs at random with no clearly associated risk factors and no family history of the disease.

"This study presents apolipoprotein B-100 as a novel therapeutic target specific for the predominant sporadic form of amyotrophic lateral sclerosis and establishes proof-of-concept to support CSF pheresis as a therapeutic strategy for mitigating neurotoxicity in sporadic amyotrophic lateral sclerosis," the study's authors state.

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